A drug approved as the first breakthrough treatment for a leading cause of blindness has left some patients with the very fate it aimed to prevent, triggering alarm over its unforeseen side effects.
The Potential Impact
The U.S. Food and Drug Administration granted approval (pdf) in February for a new drug, Syfovre (pegcetacoplan injection), to treat geographic atrophy, an advanced form of dry age-related macular degeneration that can lead to blindness. The drug was initially hailed as a breakthrough in the fight against this previously untreatable condition affecting nearly 1 in 100 Americans over 50.“The approval of Syfovre is the most important event in retinal ophthalmology in more than a decade,” Dr. Eleonora Lad, lead investigator for the OAKS study, sponsored by Apellis Pharmaceuticals, Inc., which manufactures Syfovre, and director of ophthalmology clinical research at Duke University Medical Center, said in a statement at the time.
However, excitement turned to concern when cases of severe eye inflammation and vision loss emerged after the drug’s launch. The American Society of Retinal Specialists, the world’s largest organization of retina specialists, issued a safety notice after physicians reported multiple instances of occlusive retinal vasculitis (ORV), a severe potentially blinding type of inflammation that obstructs the blood flow in the vessels that nourish the retina, Biopharma Dive reported.
Story continues below advertisement
Of the first six reported cases, five patients were left blind in the treated eye and one woman went completely blind after receiving Syfovre in both eyes.
There is an eighth suspected case of ORV that caused blindness in the treated eye, which has not yet been confirmed, according to Stat news.
The Response
These adverse events were not seen in clinical trials, raising questions about the drug’s safety profile.In response to the reported ORV cases, Apellis published a press release stating that there was no indication that “drug product or manufacturing issues” contributed to the rare but serious side effect. The drugmaker has not identified a root cause but confirmed at least seven cases of non-occlusive/occlusive among the first 68,000 patients treated since Syfovre’s launch in February.
Throughout more than 23,000 clinical trial injections, there were no reported instances of the condition, according to Appellis. No alterations were made to the drug formulation between the Phase III clinical trials and the commercial release of supplies.
The most frequent adverse reactions to Syfovre include ocular discomfort, neovascular age-related macular degeneration (wet AMD), vitreous floaters, and conjunctival hemorrhage, as per the prescribing information (pdf).
Following the report of the severe adverse effect, Apellis revealed data from the an extension study for Syfovre, which includes 30 months of continuous treatment. The data “reinforces” the long-term efficacy and safety of the drug, according to the company.
Copy
